# Topical chemotherapy in cutaneous T-cell lymphoma: positive results of a randomized, controlled, multicenter trial testing the efficacy and safety of a novel mechlorethamine, 0.02%, gel in mycosis fungoides

Source: https://onco.cc/key-papers/paper-study-201-jama-dermatol-2013/  
OnCo record `paper-study-201-jama-dermatol-2013` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

The primary report of Study 201: a ready-made mechlorethamine gel cleared index lesions in 58.5 percent of early mycosis fungoides patients against 47.7 percent with the traditional compounded ointment, meeting the noninferiority bar.

## Summary

Randomised, controlled, observer-blinded, multicentre trial at academic medical and cancer centres comparing mechlorethamine hydrochloride 0.02% gel with mechlorethamine 0.02% compounded ointment, applied once daily for up to 12 months, in 260 patients with stage IA to IIA mycosis fungoides who had not used topical mechlorethamine within 2 years and were naive to topical carmustine. The primary endpoint was response by the Composite Assessment of Index Lesion Severity (CAILS); secondary endpoints were the Modified Severity-Weighted Assessment Tool and time-to-response analyses.

Response rates for gel versus ointment were 58.5 versus 47.7 percent by CAILS and 46.9 versus 46.2 percent by the Modified Severity-Weighted Assessment Tool. The ratio of gel to ointment CAILS response rates was 1.23 (95% CI 0.97 to 1.55), which met the prespecified criterion for noninferiority. Time-to-response analyses demonstrated superiority of the gel.

## Fields

- Kind: Key paper
- Last checked: 2026-09-24
- Journal: JAMA Dermatology
- Year: 2013
- DOI: 10.1001/2013.jamadermatol.541
- Authors: Lessin SR, Duvic M, Guitart J, et al.
- Findings: CAILS response 58.5% with mechlorethamine 0.02% gel vs 47.7% with compounded ointment; ratio 1.23 (95% CI 0.97 to 1.55), noninferiority met.; Modified Severity-Weighted Assessment Tool response 46.9% vs 46.2%.; Time-to-response analyses favoured the gel.
- What it means: This trial gave mechlorethamine, the first chemotherapy drug ever used, a modern licensed form: the FDA approved Valchlor gel in 2013 for early mycosis fungoides-type cutaneous T-cell lymphoma after skin-directed therapy, replacing pharmacy-compounded preparations of uncertain stability.
- Caveats: Active-controlled noninferiority design against a compounded ointment; no placebo or untreated arm.; Observer-blinded rather than double-blinded for the applied product.; The label's efficacy table excludes 18 patients with randomisation protocol violations and reports 60% vs 48%.

## Sources

- JAMA Dermatology 2013: https://doi.org/10.1001/2013.jamadermatol.541
- PubMed: https://pubmed.ncbi.nlm.nih.gov/23069814/
- ClinicalTrials.gov NCT00168064: https://clinicaltrials.gov/study/NCT00168064

## Connected records

- cancers: [Cutaneous T-cell lymphoma (mycosis fungoides and Sezary syndrome)](https://onco.cc/cancers/cutaneous-t-cell-lymphoma/)
- drugs: [Mechlorethamine (chlormethine)](https://onco.cc/drugs/mechlorethamine/)
- companies: [Helsinn](https://onco.cc/companies/helsinn/)
- trials: [Study 201](https://onco.cc/trials/study-201/)

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